Impact of Imetelstat Treatment on PROs and Health Care Resource Utilization in Heavily Transfused Non-Del(5Q) Lower-Risk Myelodysplastic Syndromes Relapsed/Refractory to Erythropoiesis Stimulating Agents: IMerge Phase 3 Trial
Author(s)
Sekeres M1, Santini V2, Diez-Campelo M3, Komrokji R4, Fenaux P5, Savona M6, Madanat Y7, Valcárcel-Ferreiras D8, Illmer T9, Jonášová A10, Bělohlávková P11, Regnault A12, Creel K13, Sengupta N14, Sherman L14, Berry T14, Dougherty S14, Shah S14, Xia Q14, Sun L14, Wan Y14, Huang F14, Ikin A14, Navada S14, Feller F14, Zeidan A15, Platzbecker U16
1Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA, 2Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Tuscany, Italy, 3The University Hospital of Salamanca, Salamanca, Castile-León, Spain, 4Moffitt Cancer Center, Tampa, FL, USA, 5Hôpital Saint-Louis, Université de Paris, Paris, Île-de-France, France, 6Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, Nashville, TN, USA, 7Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX, USA, 8Hospital Universitari Vall d'Hebron, Barcelona, Spain, 9Hematology Private Practice, Dresden, Saxony, Germany, 10General Hospital, Prague, Bohemia, Czech Republic, 11Charles University Hospital, Hradec Kralove, Bohemia, Czech Republic, 12Modus Outcomes, a division of THREAD, Lyon, 69, France, 13Modus Outcomes, a division of THREAD, Cambridge, MA, USA, 14Geron Corporation, Parsippany, NJ, USA, 15Yale School of Medicine and Yale Cancer Center, Yale University, New Haven, CT, USA, 16Leipzig University Hospital, Leipzig, Saxony, Germany
Presentation Documents
OBJECTIVES: Imetelstat, a first-in-class telomerase inhibitor, showed high rates of durable transfusion independence (TI) across all lower-risk myelodysplastic syndrome (LR-MDS) patient subtypes in the phase 3 IMerge trial. Here, the impact of imetelstat on patient-reported outcomes (PROs) and health care resource utilization (HCRU) was explored.
METHODS: PRO data were collected using the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and EQ-5D-5L. HCRU data were collected throughout the trial.
RESULTS: PRO population included 118 and 57 patients receiving imetelstat and placebo, respectively. Mean age was 71 years (range, 39–87 years); 62.3% of patients were men. Overall, 50.0% of imetelstat-treated vs 40.4% of placebo-treated patients reported sustained meaningful improvement in fatigue (defined as ≥3-point increase on the FACIT-Fatigue score). Median time to first sustained meaningful improvement in fatigue was shorter with imetelstat than placebo (28.3 vs 65.0 weeks; hazard ratio, 1.34; 95% CI, 0.82–2.20.) After 12 weeks, more imetelstat-treated than placebo-treated patients reported improvement in fatigue. In the imetelstat group, a higher proportion of responders had sustained meaningful improvement in fatigue scores consistently vs nonresponders across 8- and 24-week TI (primary and secondary end points); this association was not observed among patients receiving placebo. Average EQ-5D-5L index scores (UK value set) through cycle 30 were 0.75 with imetelstat vs 0.69 with placebo. Despite limitations in outpatient HCRU data (36.4% imetelstat vs 40.0% placebo), results of further analysis showed a shorter length of hospital stay with imetelstat vs placebo (median, 6 vs 25.5 days).
CONCLUSIONS: Fatigue associated with anemia is the most debilitating symptom experienced by patients with transfusion-dependent LR-MDS. IMerge is the first randomized global trial, in which patients with a transfusion burden of ≥4 U showed an improvement in PRO and a reduction of HCRU when treated with imetelstat.
Conference/Value in Health Info
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
CO77
Topic
Clinical Outcomes, Patient-Centered Research, Study Approaches
Topic Subcategory
Clinical Outcomes Assessment, Clinical Trials, Comparative Effectiveness or Efficacy, Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas